Stem Cell News

Seeking a Cure for Retinal Disease

$10.6 Million New York State Grant Would EnableNSCI-Discovered Retinal Stem Cell to Prepare for Clinical Trial

RENSSELAER, N.Y. (December 12, 2012) — The Regenerative Research Foundation (RRF), the not-for-profit organization that has the Neural Stem Cell Institute (NSCI) as its research arm and is the hub of the Retinal Stem Cell Consortium, has been recommended for a grant of $10.6 million by the Funding Committee of the Empire State Stem Cell Board (ESSCB) and the NYS stem cell program, NYSTEM.

The award would be divided with $5.8 going to the RRF and $4.8 distributed to the other members of the Retinal Stem Cell Consortium over a 4-year period to enable them to take NSCI’s discovery of the RetinalPigment Epithelium Stem Cell (RPESC) to clinical trial for Age-Related Macular Degeneration (AMD), the leading cause of blindness that affects 1 out of 5 people over age 75.

Researchers at the NSCI, led by Dr. Sally Temple and Dr. Jeffrey Stern — co-founders of the RRF, discovered this new RPESC adult stem cell source, present at the back of the human eye. These RPESCs are silent or dormant inside the normal adult eye, but once extracted they activate and behave as stem cells, capable of making stable, healthy RPE cells.

“The Retinal Pigment Epithelium is a film of tissue at the back of the eye that degenerates in patients with Age-related Macular Degeneration, robbing them of vision,” said Dr. Temple.

“This is a critical investment for the Neural Stem Cell Institute,” said U.S. Senator Kirsten Gillibrand.” I saw first-hand the research being done at this facility where science is leading to new and exciting discoveries that are producing clear results. With the necessary resources, scientists can continue to get closer to unlocking the complex applications of stem cell research. With these funds we may be one step closer to finding a cure for Age-Related Macular Degeneration.”

NSCI created the Retinal Stem Cell Consortium to generate stem-cell-based therapies to treat blinding retinal disease. The consortium consists of world-leading stem cell scientists, retinal researchers, ophthalmologists and representatives of biotechnology companies, working together to create a seamless pathway from the laboratory to the clinic.

This consortium will perform the work necessary to apply to the U.S. Food and Drug Administration (FDA) to allow a Phase 1 clinical trial using the RPESC as a cell transplant therapy for AMD. This includes making clinical grade cells, which will be performed at a NYSTEM funded core facility at the University of Rochester, and demonstrating their functionality, efficacy and safety.

Upon successful completion of the project goals, the consortium will have accelerated the RPESC towards the clinic. This work will enable a clinical trial to occur. “If RPESC-based cell transplantation proves to be a valuable therapy, this will be of great benefit to AMD patients and families. Moreover, the treatment can be provided at New York State medical centers, allowing our large AMD population to access the new therapy close to home,” said Dr. Jeffrey Stern, chairman of RRF.

There are 11 members of the retinal stem cell consortium, including the RRF. They are: the University of Rochester, Fordham University, General Electric, Rensselaer Polytechnic Institute, Health Research, Inc./Wadsworth Center, Columbia University, National Eye Institute, University of Pittsburgh and the University at Albany.

The consortium program is a new initiative of NYSTEM, a program administered by the Department of Health that is playing a key role to ensure NYS is at the leading edge of stem cell research. NYSTEM’s mission is to accelerate the growth of scientific knowledge about stem cell biology and the development of therapies and diagnostic methods under the highest ethical, scientific, and medical standards for the purpose of alleviating disease and improving human health.

“This exciting NSCI project is part of NYSTEM’s boldest initiative to date and one of three awards approved by the Funding Committee of the Empire State Stem Cell Board,” State Health Commissioner Nirav R. Shah, M.D., M.P.H. said. “Like Dr. Temple’s and Dr. Stern’s promising effort to develop a treatment for Age-related Macular Degeneration, these projects each target a specific disease and demonstrate real hope of being in human clinical trials within five years.”

UAlbany Vice President for Research James Dias said, “The University at Albany is playing a critical role in helping private startups and institutes develop pipelines for therapeutics by providing high tech, cutting edge resources and an outstanding infrastructure here on the UAlbany East Campus. The Neural Stem Cell Institute is developing advanced therapies for macular degeneration and UAlbany is proud to provide the infrastructure in which they operate.”

About the Neural Stem Cell Institute

The Neural Stem Cell Institute (NSCI) it is the only independent, non-profit stem cell research institute in the United States. Headquartered in Rensselaer, it is the research arm of the non-profit Regenerative Research Foundation (RRF). It is a unique organization that produces leading stem cell research to develop new therapies for diseases of the central nervous system (CNS). NSCI aims to harness the power of stem cells to ease suffering caused by injury and disease of the brain, spinal cord and retina.

NSCI was co-founded by MacArthur Award winner, Dr. Sally Temple of Slingerlands, who helped discover and define nervous system stem cells. A native of York, England, she has lived for over 20 years in the Albany area and leads a team of researchers focused on using neural stem cell therapies for brain, eye and spinal cord disorders including Alzheimer’s Disease, Age-related Macular Degeneration, Spinal cord injury, Optic nerve injury, Multiple Sclerosis and Parkinson’s Disease.

Our Unique Organization

The Neural Stem Cell Institute (NSCI) is dedicated to developing regenerative stem cell therapies for various diseases of the central nervous system (CNS): the brain, spinal cord and retina, regardless of the cause of injury. Led by MacArthur “Genius” Award winner Dr. Sally Temple who helped discover and define nervous system stem cells, NSCI is the first independent, non-profit stem cell research institute in the USA.